北京SeladelparIV期临床试验-Seladelpar治疗原发性胆汁性胆管炎（PBC）伴代偿期肝硬化患者的研究

登记号	CTR20253298	试验状态	进行中
申请人联系人	裘桂潇	首次公示信息日期	2025-08-15
申请人名称	Gilead Sciences, Inc./ 吉利德（上海）医药科技有限公司/ Lonza Tampa LLC

登记号	CTR20253298
相关登记号	暂无
药物名称	Seladelpar
药物类型	化学药物
临床申请受理号	企业选择不公示
适应症	原发性胆汁性胆管炎（PBC）伴代偿性肝硬化
试验专业题目	AFFIRM：一项评价 Seladelpar 对原发性胆汁性胆管炎 (PBC) 伴 代偿性肝硬化患者的临床结局的影响的随机、双盲、安慰剂对照研究
试验通俗题目	Seladelpar治疗原发性胆汁性胆管炎（PBC）伴代偿期肝硬化患者的研究
试验方案编号	CB8025-41837	方案最新版本号	3.1（中国）
版本日期:	2025-01-28	方案是否为联合用药	否

申请人名称	[["Gilead Sciences, Inc."],["吉利德（上海）医药科技有限公司"],["Lonza Tampa LLC"]]
联系人姓名	裘桂潇	联系人座机	021-22276880	联系人手机号
联系人Email	Guixiao.Qiu2@gilead.com	联系人邮政地址	上海市-上海市-上海市	联系人邮编	200122

目的: 评价 Seladelpar 与安慰剂相比对 EFS 的影响。EFS 定义为从治疗开始至首 次发生以下任何裁定事件（直至第 156 周）的时间： ？ 任何原因导致的死亡 ？ 肝移植 ？ MELD 评分 ≥ 15（见附录 I) ？ 需要治疗的腹水 ？ 因符合以下任一条件的事件而住院治疗： i. 食管或胃静脉曲张出血 ii. 肝性脑病（定义为 West Haven 评分 ≥ 2，附录 H) iii. 自发性细菌性腹膜炎，通过诊断性腹腔穿刺获得的 WBC 计数、分类计数和/ 或需氧和/或厌氧血培养瓶出现阳性生长所证实

试验分类	安全性和有效性	试验分期	IV期	设计类型	平行分组
随机化	随机化	盲法	双盲	试验范围	国际多中心试验

年龄	18岁(最小年龄)至 无上限 (最大年龄)
性别	男+女
健康受试者	无
入选标准	[["年龄必须至少为 18 岁"],["必须确诊患有 PBC"],["有肝硬化的证据"],["CP 评分为 A 级或 B 级"],["具有生育能力的有性生活的女性必须在研究期间以及末次给药后至少 90 天内使用至少 1 种屏障避孕法和另一种有效的避孕方法。与具有生育能力的女 性伴侣保持性生活的男性受试者必须在研究期间以及末次给药后至少 90 天内使用 屏障避孕法，同时其女性伴侣必须使用另一种有效的避孕方法"]]
排除标准	[["曾暴露于 Seladelpar"],["存在 PBC 以外的其他医疗状况，研究者认为会导致受试者无法完整参与本研究"],["有肝移植病史或被列入尸体移植或计划的活体供体移植的活跃名单"],["失代偿性肝硬化"],["筛选期经多普勒超声成像，或既往 CT 或 MRI 显示有门静脉血栓形成的证据"],["筛选前 12 周内因肝脏相关并发症而住院治疗"],["筛选期间的实验室参数： a. ALP < 1.5× ULN 或 ≥ 10× ULN b. ALT 或 AST ≥ 5x ULN c. TB ≥ 5× ULN d. 血小板计数 ≤ 50×103 /μL e. 白蛋白 ≤ 2.8 g/dL f. 肾小球滤过率估计值 (eGFR) < 45 mL/min/1.73 m2 g. MELD 评分 > 12。对于接受抗凝药物治疗的受试者，在确定用于计算 MELD 评分的基线 INR 时，应咨询申办方的医学监查员，将抗凝剂的使用情况考虑 在内。 h. 血清甲胎蛋白 (AFP) > 20 ng/mL i. INR > 1.7"],["CP-C 级肝硬化"],["合并有其他肝脏疾病"]]

试验药	[["中文通用名:Seladelpar 英文通用名:Seladelpar 商品名称:Livdelzi","剂型:胶囊 规格:5mg/粒、10mg/粒 用法用量:伴CP-A级肝硬化的受试者将以10mg每日一次的剂量开始给药；伴CP-B级肝硬化的受试者将以5mg每日一次的剂量开始给药 用药时程:将在每天大致同一时间口服给药，每日一次，持续最长不超过156周（3年）。"]] 序号 名称 用法
对照药	[["中文通用名:安慰剂 英文通用名:NA 商品名称:NA","剂型:胶囊 规格:0mg/粒 用法用量:伴CP-A或CP-B级肝硬化的受试者将每日一次服用安慰剂 用药时程:将在每天大致同一时间口服给药，每日一次，持续最长不超过156周（3年）。"]] 序号 名称 用法

主要终点指标及评价时间	[["无事件生存期","36个月","有效性指标"]] 序号 指标 评价时间 终点指标选择
次要终点指标及评价时间	[["总生存期","36个月","有效性指标"],["无肝移植生存期","36个月","有效性指标"],["至因符合任何条件的裁定事件而住院治疗的时间","36个月","有效性指标"],["至每起其他裁定 PBC 临床事件发生的时间","36个月","有效性指标"]] 序号 指标 评价时间 终点指标选择

1	姓名	贾继东	学位	博士后	职称	正高级
	电话	13501378269	Email	jia_jd@ccmu.edu.cn	邮政地址	北京市-北京市-西城区永安路95号
	邮编	100050	单位名称	首都医科大学附属北京友谊医院

[["首都医科大学附属北京友谊医院","贾继东","中国","北京市","北京市"],["四川大学华西医院","杨丽","中国","四川省","成都市"],["浙江大学医学院附属第一医院","杨益大","中国","浙江省","杭州市"],["首都医科大学附属北京地坛医院","李明慧","中国","北京市","北京市"],["南京市第二医院","杨永峰","中国","江苏省","南京市"],["云南省第一人民医院","耿嘉蔚","中国","云南省","昆明市"],["河南省人民医院","尚佳","中国","河南省","郑州市"],["首都医科大学附属北京佑安医院","徐斌","中国","北京市","北京市"],["新疆医科大学第一附属医院","鲁晓擘","中国","新疆维吾尔自治区","乌鲁木齐市"],["西安交通大学第一附属医院","何英利","中国","陕西省","西安市"],["福建医科大学孟超肝胆医院","黄祖雄","中国","福建省","福州市"],["贵州省人民医院","罗新华","中国","贵州省","贵阳市"],["重庆医科大学附属第二医院","蔡大川","中国","重庆市","重庆市"],["华中科技大学同济医学院附属协和医院","蔺蓉","中国","湖北省","武汉市"],["吉林大学第一医院","高沿航","中国","吉林省","长春市"],["上海交通大学医学院附属仁济医院","马雄","中国","上海市","上海市"],["中山大学附属第三人民医院","林炳亮","中国","广东省","广州市"],["西京医院","韩英","中国","陕西省","西安市"],["镇江市第三人民医院","谭友文","中国","江苏省","镇江市"],["安徽医科大学第一附属医院","郜玉峰","中国","安徽省","合肥市"],["Buenos Aires Macula Clinical Research (BAM Clinical Research)","Raul ,Adrover","阿根廷","NA","Ciudad Autónoma de Buenos Aires"],["CIPREC (Centro de Investigacion y Prevencion Cardiovascular)","Fernando ,Gruz","阿根廷","NA","Ciudad Autónoma de Buenos Aires"],["Alfred Health","Stuart ,Roberts","澳大利亚","VIC","Melbourne"],["Flinders Medical Centre","Kate ,Muller","澳大利亚","SA","Adelaide"],["GI Research Institute","Hin Hin ,Ko","加拿大","BC","Vancouver"],["Centro Clinico Mediterraneo","Carlos ,Bresky Ruiz","智利","NA","La Serena"],["Pontificia Universidad Católica de Chile","Carlos Esteban ,Benítez Gajardo","智利","NA","Santiago"],["Biocinetic","Elizabeth ,Arriagada Hernández","智利","NA","Santiago"],["Clinical Research Chile SpA.","Alex ,Ruiz Salas","智利","NA","Valdivia"],["Yonsei University Severance Hospital","Seung Up ,Kim","韩国","NA","Seoul"],["Seoul National University Bundang Hospital","Sook-Hyang ,Jeong","韩国","NA","Seongnam"],["Kyungpook National University Hospital","Won Young ,Tak","韩国","NA","Daegu"],["Pusan National University Hospital","Jeong ,Heo","韩国","NA","Busan"],["Marmara University Pendik Training and Research Hospital","Coskun Ozer ,Demirtas","土耳其","NA","Pendik"],["Adana City Training and Research Hospital","Hilmi Erdem ,Sumbul","土耳其","NA","Adana"],["Rize Recep Tayyip Erdogan University Training and Research Hospital","Yusuf ,Yilmaz","土耳其","NA","Rize Merkez"],["Ankara City Hospital","Meral ,Akdogan","土耳其","NA","Ankara"],["Katip Celebi University- Izmir Ataturk Egitim ve Arastirma Hastanesi","Sezgin ,Vatansever","土耳其","NA","Karaba？lar"],["Ege Universitesi Tip Fakultesi","Ulus ,Akarca","土耳其","NA","Izmir"],["Mersin University Medical","Fehmi ,Ates","土耳其","NA","Mersin"],["Gaziantep University Medical Faculty Sahinbey Research and Practice Hospital","Taylan ,Metin","土耳其","NA","Gaziantep"],["Kocaeli University Hospital","Hasan ,Yilmaz","土耳其","NA","Kocaeli"],["Uludag University Medical Faculty Hosp.","Murat ,Kiyici","土耳其","NA","Gorukle"],["University of Miami","Cynthia ,Levy","美国","FL","Miami"],["Liver Institute Northwest","Kris V. ,Kowdley","美国","WA","Seattle"],["Henry Ford Health System","Stuart C. ,Gordon","美国","MI","Detroit"],["University of California Davis","Christopher ,Bowlus","美国","CA","Sacramento"],["Atrium Health Wake Forest Baptist","Herbert ,Bonkovsky","美国","NC","Winston Salem"],["University of Iowa Hospitals and Clinics","Alan ,Gunderson","美国","IA","Iowa City"],["University of Chicago","Kapuluru Gautham ,Reddy","美国","IL","Chicago"],["University of Pittsburgh School of Medicine","Mordechai ,Rabinovitz","美国","PA","Pittsburgh"],["Northwell Health","Sanjaya ,Satapathy","美国","NY","Manhasset"],["Virginia Commonwealth University","Velimir ,Luketic","美国","VA","Richmond"],["University of Louisville","Matthew ,Cave","美国","KY","Louisville"],["Thomas Jefferson University","David ,Sass","美国","PA","Philadelphia"],["Ochsner Clinic Foundation","Shobha ,Joshi","美国","LA","Jefferson"],["Piedmont Healthcare (GA)","Parita ,Patel","美国","GA","Atlanta"],["University of California San Francisco","Jennifer ,Lai","美国","CA","San Francisco"],["Om Research LLC","Jatinder ,Pruthi","美国","CA","Lancaster"],["MedStar Georgetown University Hospital","Coleman ,Smith","美国","VA","Fairfax"],["Icahn School of Medicine at Mount Sinai","Joseph ,Odin","美国","NY","New York"],["California Pacific Medical Center","Kidist ,Yimam","美国","CA","San Francisco"],["Tulane University","Peng-Sheng ,Ting","美国","LA","New Orleans"],["Cedars Sinai Medical Center","Walid ,Ayoub","美国","CA","Los Angeles"],["Covenant Research and Clinics, LLC","Guy ,Neff","美国","FL","Fort Myers"],["Gastroenterology Center of the MidSouth, P.C","Ziad ,Younes","美国","TN","Cordova"],["Bon Secours St. Mary's Hospital of Richmond","Mitchell L. ,Shiffman","美国","VA","Newport News"],["University of Texas Southwestern Medical Center","Marlyn ,Mayo","美国","PA","Hershey"],["Covenant Metabolic Specialists","Guy ,Neff","美国","TX","Dallas"],["California Liver Research Institute","Edward ,Mena","美国","CA","Pasadena"],["UH Cleveland Medical Center","Anthony ,Post","美国","OH","Cleveland"],["Kaiser Permanente","Amandeep ,Sahota","美国","CA","Los Angeles"],["NYU Langone Hospital","Ira M. ,Jacobson","美国","NY","New York"],["Mercy Medical Center","Madhavi ,Rudraraju","美国","MD","Baltimore"],["Beth Israel Deaconess Medical Center","Paul J. ,Thuluvath","美国","MA","Boston"],["Massachusetts General Hospital","Daniel ,Pratt","美国","MA","Boston"],["Liver Institute at Methodist Dallas","Mangesh ,Pagadala","美国","TX","Dallas"],["Florida Digestive Health Specialists","Manuel ,Rodriguez","美国","FL","Tampa"],["Penn State Milton S. Hershey Cancer Institute","Karen ,Krok","美国","PA","Hershey"],["Pinnacle Clinical Research, PLLC","Madhavi ,Rudraraju","美国","TX","San Antonio"]]

序号	机构名称	主要研究者	国家	省（州）	城市

[["首都医科大学附属北京友谊医院 生命伦理委员会伦理","同意","2025-03-12"]]

序号	名称	审查结论	批准日期/备案日期

进行中（尚未招募）

目标入组人数	国内: 40 ； 国际: 318 ；
已入组人数	国内: 登记人暂未填写该信息； 国际: 78 ；
实际入组总人数	国内: 登记人暂未填写该信息； 国际: 登记人暂未填写该信息；

第一例受试者签署知情同意书日期	国内：登记人暂未填写该信息；     国际：2023-09-07；
第一例受试者入组日期	国内：登记人暂未填写该信息；     国际：2023-10-19；
试验完成日期	国内：登记人暂未填写该信息；     国际：登记人暂未填写该信息；